Novartis Receives Third FDA Breakthrough Designation

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FDA has granted Novartis breakthrough therapy designation to BYM338 for sporadic inclusion body myositis.

The US Food and Drug Administration (FDA) has granted Novartis breakthrough therapy designation—the company’s third such designation this year—to BYM338 for sporadic inclusion body myositis (sIBM).

FDA crated the breakthrough therapy designation to expedite the development and review of new drugs for serious or life-threatening conditions. The designation requires preliminary clinical evidence that demonstrates substantial improvement over currently available therapy. The designation includes all of the fast track program features, as well as more intensive FDA interaction and guidance.

Novartis reports that the breakthrough designation is based on the results of a Phase II proof-of-concept study that showed BYM338 substantially benefited patients with sIBM compared to placebo. The results of this study will be presented at the American Neurological Association meeting on Oct. 14 and is expected to be published in a major medical journal later this year, according to a company statement.

sIBM is a rare, yet potentially life-threatening muscle-wasting condition. Patients who have the disease can gradually lose the ability to walk, experience falls and injuries, lose hand function, and have swallowing difficulties.

"BYM338 is the third example this year of Novartis' leadership in bringing breakthrough therapies to patients reinforcing our commitment to innovation addressing significant unmet medical needs and enhancing the lives of patients," said Timothy Wright, M.D., Global Head of Development, Novartis Pharmaceuticals. "With no effective therapies currently available for sIBM, bimagrumab has the potential to be the first real option for patients with this condition."


BYM338 (bimagrumab) is a novel, fully human monoclonal antibody developed to treat pathological muscle loss and weakness. BYM338 was developed by the Novartis Institutes for Biomedical Research, in collaboration with Morphosys, whose HuCAL library was used to identify the antibody. BYM338 binds with high affinity to type II activin receptors, preventing natural ligands from binding, including myostatin and activin. BYM338 stimulates muscle growth by blocking signaling from these inhibitory molecules.

Earlier this year, Novartis received Breakthrough Therapy designation status for RLX030 (serelaxin), an investigational treatment for patients with acute heart failure (AHF) and LDK378 for the treatment of patients with anaplastic lymphoma kinase positive (ALK+) metastatic non-small cell lung cancer.


Source: Novartis